An Open-Label Extension for the Phase 2 Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS

Phase 2

Active, not recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: CNMAu8

• Registration Number: NCT05299658
• Lead Sponsor: Clene Nanomedicine

Brief Summary

This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.

Detailed Description

A forty-eight (48) week optional open-label extension period (Open- Label Period), which may be extended by 12-week increments until discontinued by the Sponsor for participants that have completed protocol CNMAu8.205.

Study Timeline

• Study Start: 2021-11-13
• Study First Submitted: 2022-03-08
• Study First Submitted QC: 2022-03-17
• Study First Posted: 2022-03-29
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-17
• Last Update Posted: 2024-07-19
• Primary Completion: 2024-11
• Study Completion: 2024-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Participants must have completed the randomized placebo-controlled Treatment Period without compliance issues.
2. Able to understand and give written informed consent to participate in the open-label extension.
3. If referred from a third party (neurologist or a State based ALS organisation), participant agrees to maintain transfer of care to a neurologist participating in the study.

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Exclusion Criteria

1. Lack of treatment compliance during the randomized placebo controlled Treatment Period.
2. Positive pregnancy test at the Week 36 visit, or, females who plan to get pregnant during the course of this extension or within 6 months of the end of this extension.
3. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.
4. Patient with clinically significant abnormalities in haematology, blood chemistry, ECG, or physical examination identified during the W36 visit which according to Investigator may interfere with continued participation.
5. Patients with clinically significant hepatic or renal dysfunction or clinical laboratory findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (< 150 x 10^9 per liter) or eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter) at the Week 36 visit.
6. Patient is considered a suicide risk in the opinion of the Investigator, has previously made a suicide attempt, or is currently demonstrating active suicidal ideation. Subjects with intermittent passive suicidal ideation are not necessarily excluded based on the assessment of the Investigator.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open label extension	CNMAu8	This is an unblinded open-label extension, all participants will be on active drug.

Primary Outcome Measures

Name	Time	Method
Electromyography measures	Up to an estimated 96 weeks.	Mean change in the average difference between active treatment and placebo from Baseline through End of Study for the MUNIXscore(4), which is the sum of the respective MUNIX values for the Abductor Digiti Minimi (ADM), Abductor Pollicis Brevis (APB), Biceps Brachii (BB), and Tibialis Anterior (TA).
Safety measures	Up to an estimated 96 weeks.	Safety endpoints include incidence of treatment-emergent AEs, drug-related AEs, deaths, SAEs, and AEs leading to discontinuation from the study. Changes from baseline (Week 36 of the placebo controlled phase) in clinical laboratory results, physical examination findings, vital signs, ECGs, and C-SSRS will be summarized descriptively by group and timepoint.

Trial Locations

Locations (2)

Neuroscience Research Australia; 🇦🇺 Randwick, New South Wales, Australia

Concord Hospital; 🇦🇺 Concord, New South Wales, Australia